F-Prime's Ben Aus­pitz co-leads $30M Se­ries A with Or­biMed for his lat­est rare dis­ease ven­ture

Hav­ing spent his 13 years at F-Prime Part­ners back­ing and ad­vis­ing rare dis­ease com­pa­nies — in­clud­ing Di­men­sion Ther­a­peu­tics and Or­chard Ther­a­peu­tics, which he co-found­ed — Ben Aus­pitz is in the game again with a brand new biotech start­up.

Ben Aus­pitz

Co-lead­ing a $30 mil­lion Se­ries A round for Modis Ther­a­peu­tics with Or­biMed, Aus­pitz is jump­ing in as board chair­man. As for the CEO post, F-Prime has tapped one of its en­tre­pre­neurs-in-res­i­dence, Joshua Grass, who’s al­so a 15-year BD vet­er­an at Bio­Marin.

Head­quar­tered in Oak­land, CA with of­fices in New York City, Modis has a clear­ly de­fined fo­cus on mi­to­chon­dr­i­al DNA de­ple­tion dis­or­ders, a group of ge­net­ic dis­eases that could af­fect the brain, mus­cles and any num­ber of or­gans.

Mi­chio Hi­ra­no of Co­lum­bia Uni­ver­si­ty Irv­ing Med­ical Cen­ter and Ra­mon Martí at Barcelona’s Vall d’He­bron Re­search In­sti­tute are cred­it­ed for Modis’ lead ther­a­py, MT1621, which treats the thymi­dine ki­nase 2 de­fi­cien­cy (TK2d), a po­ten­tial­ly fa­tal mus­cle-weak­en­ing dis­ease. The drug it­self is mix of nu­cle­o­sides that the re­searchers be­lieve can ad­dress the cause of mi­to­chon­dr­i­al dys­func­tion and has shown to pro­long sur­vival in an­i­mal mod­els.

Col­lab­o­ra­tors in Spain have be­gun to test MT1621 in hu­mans, too, with “en­cour­ag­ing re­sults.”

Joshua Grass

“This is a com­pelling or­phan drug de­vel­op­ment op­por­tu­ni­ty for a po­ten­tial­ly high im­pact ther­a­peu­tic for a very se­ri­ous dis­ease that af­fects both chil­dren and adults,” said CEO Grass. “The in­creas­ing use of ad­vanced DNA se­quenc­ing tech­nol­o­gy to di­ag­nose pa­tients with mi­to­chon­dr­i­al dis­ease at the ge­net­ic lev­el cre­ates new op­por­tu­ni­ties to tar­get ther­a­peu­tics to spe­cif­ic caus­es of dis­ease with large treat­ment ben­e­fits.”

Rishi Gup­ta

To ful­ly cap­ture that op­por­tu­ni­ty, Modis is work­ing on oth­er mol­e­cules and ther­a­peu­tic modal­i­ties to treat TK2d as well as oth­er ge­net­i­cal­ly-de­fined mi­to­chon­dr­i­al dis­eases.

Found­ing in­vestor Ac­eras Life Sci­ences al­so par­tic­i­pat­ed in the round, with Os­age Uni­ver­si­ty Part­ners tag­ging along. Or­biMed part­ner Rishi Gup­ta is join­ing the board.

Paul Hudson, Getty Images

UP­DAT­ED: Sanofi CEO Hud­son lays out new R&D fo­cus — chop­ping di­a­betes, car­dio and slash­ing $2B-plus costs in sur­gi­cal dis­sec­tion

Earlier on Monday, new Sanofi CEO Paul Hudson baited the hook on his upcoming strategy presentation Tuesday with a tell-tale deal to buy Synthorx for $2.5 billion. That fits squarely with hints that he’s pointing the company to a bigger future in oncology, which also squares with a major industry tilt.

In a big reveal later in the day, though, Hudson offered a slate of stunners on his plans to surgically dissect and reassemble the portfoloio, saying that the company is dropping cardio and diabetes research — which covers two of its biggest franchise arenas. Sanofi missed the boat on developing new diabetes drugs, and now it’s pulling out entirely. As part of the pullback, it’s dropping efpeglenatide, their once-weekly GLP-1 injection for diabetes.

“To be out of cardiovascular and diabetes is not easy for a company like ours with an incredibly proud history,” Hudson said on a call with reporters, according to the Wall Street Journal. “As tough a choice as that is, we’re making that choice.”

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De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

What does $6.9B buy these days in on­col­o­gy R&D? As­traZeneca has a land­mark an­swer

Given the way the FDA has been whisking through new drug approvals months ahead of their PDUFA date, AstraZeneca and their partners Daiichi Sankyo may not have to wait until Q2 of next year to get a green light on trastuzumab deruxtecan (DS-8201).

The pharma giant this morning played their ace in the hole, showing off why they were willing to commit to a $6.9 billion deal — with $1.35 billion in a cash upfront — to partner on the drug.

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FDA in-house re­view spot­lights an is­sue with one of Hori­zon's end­points but notes ef­fi­ca­cy for lead drug

The FDA in-house review highlights a disagreement of investigators’ use of a key endpoint by Horizon Pharma in the late-stage trial for the top drug in its pipeline, but largely agreed that the antibody was effective.

Horizon submitted a BLA for thyroid eye disease (TED) drug teprotumumab in March, less than two years after they bought the drug (and the rest of a division) from Narrow River for $145 million upfront. With breakthrough status, priority review, orphan designation and in-house sales projections of up to $750 million, the one-time Roche reject became the marquee pipeline asset for a company that’s developed some of the world’s most expensive drugs.

Seat­tle Ge­net­ics de­tails pos­i­tive OS and PFS da­ta for tu­ca­tinib in breast can­cer

Seattle Genetics $SGEN is showing off more positive data around tucatinib, its pivotal-stage drug for HER2 positive breast cancer.

A month after hearing about solidly upbeat hazard ratios, we learned today that the estimated progression-free survival rate at one year was 33% in the tucatinib arm compared to 12% for patients taking trastuzumab and capecitabine alone.

Median PFS was 7.8 months (95% CI: 7.5, 9.6) in the tucatinib arm, compared to 5.6 months (95% CI: 4.2, 7.1) in the control arm.

Bat­tered, cash hun­gry In­tec feels the burn of No­var­tis re­jec­tion

It’s a case of some bad timing for Intec.

Just when a key trial testing the company’s Accordion drug delivery tech imploded in Parkinson’s disease, they handed Novartis data from a successful PK study of a custom Accordion pill engineered to deliver a Novartis compound to entice the Swiss drugmaker into signing a licensing agreement.

Novartis said thanks, but no thanks.

For the cash-strapped Israeli drug developer, the failure to clinch the deal marks a big blow. As of the third quarter, the company has $15.7 million in cash and equivalents, which HC Wainwright analysts estimate will keep the lights on into mid-2020.

Bris­tol-My­ers shows off a low-pro­file AML con­tender it gained from Cel­gene buy­out — and they’re tak­ing it straight to the FDA

Bristol-Myers Squibb reaped an enormous pipeline with its much-criticized $64 billion megadeal to buy Celgene. And it got a few hidden gems in the deal.

One of those gems was brought out for display on Tuesday, with a late-breaker at ASH on CC-486, which is now being prepped for regulatory filings at the FDA and elsewhere.

Celgene top-lined the positive results in a maintenance setting for acute myeloid leukemia a few months ago, but at ASH investigators pulled back the curtains on the all-important data they believe will give them an advantage in the commercial wars to come.

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De­sert­ed by Astel­las and Mer­ck, lit­tle Cor­re­vio still can't win over FDA pan­el con­cerned with its AFib drug's safe­ty

When the FDA spurned Astellas’ pitch for atrial fibrillation drug vernakalant in 2008, regulators made it abundantly clear that it wasn’t the efficacy they had a problem with — two Phase III trials had shown the drug successfully restored 52% of patients’ heartbeat from irregular to normal — but the cardio safety issues for a drug that was to compete with well established, low-risk options. One licensing deal, one clinical hold and several studies later, the chances of approval aren’t looking any better.

New trade deal knocks out long-sought bi­o­log­ics pro­tec­tions for drug­mak­ers

House Democrats negotiating with the Trump Administration on a new North American trade deal settled early on four issues: enforcement, labor and environmental standards and drug pricing.

On drug pricing, Politico reports, Trump crumbled within weeks of heightened negotiations.

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